Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety

Loss of adenosine deaminase activity leads to severe combined immunodeficiency (ADA-SCID); production and function of T, B, and natural killer (NK) cells are impaired. Gene therapy (GT) with an autologous CD34(+)-enriched cell fraction that contains CD34(+) cells transduced with a retroviral vector...

Полное описание

Сохранить в:
Библиографические подробности
Опубликовано в: :Mol Ther
Главные авторы: Cicalese, Maria Pia, Ferrua, Francesca, Castagnaro, Laura, Rolfe, Katie, De Boever, Erika, Reinhardt, Rickey R., Appleby, Jonathan, Roncarolo, Maria Grazia, Aiuti, Alessandro
Формат: Artigo
Язык:Inglês
Опубликовано: American Society of Gene & Cell Therapy 2018
Предметы:
Original Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC5910668/
https://ncbi.nlm.nih.gov/pubmed/29433935
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.12.022
Метки: Добавить метку
Нет меток, Требуется 1-ая метка записи!

Схожие документы